公开(公告)号：US20190241890A1

公开(公告)日：2019-08-08

申请号：US16127600

申请日：2018-09-11

申请人： Sarepta Therapeutics, Inc.

发明人： Patrick L. IVERSEN

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2320/12 ,  C12N2320/34

摘要： Provided herein are antisense oligonucleotides, compositions comprising antisense oligonucleotides, and methods for the use of antisense oligonucleotides in manipulating translation. Expression of isoforms of proteins expressed from different start codons of the same transcript are inhibited by antisense oligonucleotides, which may also enhance expression of non-target isoforms.

公开(公告)号：US20180255751A1

公开(公告)日：2018-09-13

申请号：US15468652

申请日：2017-03-24

申请人： The Broad Institute Inc. ,  Massachusetts Institute of Technology ,  The General Hospital Corporation

发明人： Aviv Regev ,  Oren PARNAS ,  Marko JOVANOVIC ,  Nir HACOHEN ,  Thomas EISENHAURE

IPC分类号： A01K67/027 ,  C12N15/10 ,  C12N15/85 ,  C12N15/90 ,  C12N9/22 ,  C12N7/04 ,  A61K48/00

CPC分类号： A01K67/0271 ,  A01K67/0275 ,  A01K2207/05 ,  A01K2207/12 ,  A01K2217/052 ,  A01K2217/077 ,  A01K2227/105 ,  A01K2267/0312 ,  A01K2267/0331 ,  A01K2267/0362 ,  A61K48/005 ,  C07K2319/09 ,  C12N7/04 ,  C12N9/22 ,  C12N15/1082 ,  C12N15/111 ,  C12N15/113 ,  C12N15/85 ,  C12N15/907 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2310/3513 ,  C12N2310/531 ,  C12N2320/12 ,  C12N2330/31 ,  C12N2330/51 ,  C12N2740/15043 ,  C12N2750/14143

摘要： The invention involves a method for modulating leukocyte activity, comprising delivering to a leukocyte a vector containing nucleic acid molecule(s), whereby the leukocyte contains Cas9 and the vector expresses one or more RNAs to guide the Cas9 to introduce mutations in one or more target genetic loci in the leukocyte, thereby modulating expression of one or more genes expressed in the leukocyte. The invention also involves identifying genes associated with leukocyte responses and experimental modeling of aberrant leukocyte activation and diseases associated with leukocytes by introducing mutations into leukocytes. The invention comprehends testing putative treatments with such models, e.g., testing putative chemical compounds that may be pharmaceutically relevant for treatment or gene therapy that may be relevant for treatment, or combinations thereof. The invention allows for the study of genetic diseases and putative treatments to better understand and alleviate leukocyte associated diseases.

公开(公告)号：US20180230492A1

公开(公告)日：2018-08-16

申请号：US15948785

申请日：2018-04-09

申请人： THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE

发明人： Ryan T. GILL ,  Andrew GARST

IPC分类号： C12N15/90 ,  C12N9/22 ,  C12N15/11 ,  C12N15/10

CPC分类号： C12N15/902 ,  C12N9/22 ,  C12N15/102 ,  C12N15/1024 ,  C12N15/1082 ,  C12N15/1093 ,  C12N15/11 ,  C12N15/111 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2320/11 ,  C12N2320/12 ,  C12N2330/31 ,  C40B40/08

摘要： Described herein are methods and vectors for rational, multiplexed manipulation of chromosomes within open reading frames (e.g., in protein libraries) or any segment of a chromosome in a cell or population of cells, in which various CRISPR systems are used.

公开(公告)号：US20180112218A1

公开(公告)日：2018-04-26

申请号：US15712633

申请日：2017-09-22

申请人： University of Massachusetts

发明人： Abraham L. Brass ,  Miles C. Smith

IPC分类号： C12N15/113 ,  C12Q1/18

CPC分类号： C12N15/1131 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2320/12 ,  C12Q1/18 ,  G01N33/5761 ,  G01N2500/04

摘要： Described herein are methods of identifying host factors that modulate Hepatitis B virus (HBV) replication in mammalian, e.g., human cells, as well as factors identified by those methods, and methods of treating HBV infections by targeting those factors. Zinc finger, CCHC domain containing 14 (ZCCHC14) is an exemplary host factor.

公开(公告)号：US09765334B2

公开(公告)日：2017-09-19

申请号：US12994411

申请日：2009-05-19

申请人： Moshe Hoshen ,  Raruch Brenner

发明人： Moshe Hoshen ,  Raruch Brenner

IPC分类号： C40B30/04 ,  C12Q1/68 ,  C12N15/113 ,  C12N15/11 ,  C07H21/02

CPC分类号： C12N15/1135 ,  C12N15/111 ,  C12N2310/141 ,  C12N2320/12 ,  C12Q1/6886 ,  C12Q2600/112 ,  C12Q2600/118 ,  C12Q2600/158 ,  C12Q2600/178

摘要： Described herein are compositions and methods for survival prediction in gastric cancer patients after surgical operation. The compositions are microRNA molecules associated with the prognosis of gastric cancer, as well as various nucleic acid molecules relating thereto or derived therefrom.

公开(公告)号：US20170226550A1

公开(公告)日：2017-08-10

申请号：US15501503

申请日：2015-08-04

申请人： Jianxin Ye ,  Krista Alvin ,  Siyan Zhang

发明人： Jianxin Ye ,  Krista Alvin ,  Siyan Zhang

IPC分类号： C12P21/00 ,  C12N15/113

CPC分类号： C12P21/00 ,  C12N15/111 ,  C12N15/113 ,  C12N15/85 ,  C12N2310/14 ,  C12N2310/531 ,  C12N2320/12 ,  C12P21/02

摘要： The invention provides methods and compositions for improving production of exogenous polypeptides in large scale culture of mammalian host cells. The methods and compositions utilize RNA interference to inhibit expression of one or more specific host cell proteins. The present invention is based on the identification of specific mammalian genes and sequences therein that are useful as RNAi targets to enhance the production of exogenous proteins in mammalian cell culture.

公开(公告)号：US20170175111A1

公开(公告)日：2017-06-22

申请号：US15389162

申请日：2016-12-22

申请人： President and Fellows of Harvard College ,  Trustees of Boston University

发明人： Alexander A. Green ,  Peng Yin ,  James J. Collins

IPC分类号： C12N15/11 ,  C12Q1/68

CPC分类号： C12N15/11 ,  C12N15/113 ,  C12N15/115 ,  C12N15/67 ,  C12N2310/17 ,  C12N2310/531 ,  C12N2320/11 ,  C12N2320/12 ,  C12Q1/6816 ,  C12Q1/6897 ,  C12Q2525/143 ,  C12Q2525/149 ,  C12Q2525/301

摘要： The invention provides novel and versatile classes of riboregulators, including inter alia activating and repressing riboregulators, switches, and trigger and sink RNA, and methods of their use for detecting RNAs in a sample such as a well and in modulating protein synthesis and expression.

公开(公告)号：US20170159123A1

公开(公告)日：2017-06-08

申请号：US15335271

申请日：2016-10-26

申请人： RUSH UNIVERSITY MEDICAL CENTER

发明人： Shunbin Xu

IPC分类号： C12Q1/68 ,  C12N15/113

CPC分类号： C12Q1/6883 ,  C12N15/113 ,  C12N2310/141 ,  C12N2320/12 ,  C12N2320/32 ,  C12N2330/31 ,  C12Q1/6876 ,  C12Q2600/106 ,  C12Q2600/158 ,  C12Q2600/178

摘要： Methods and compositions are disclosed to identify plasma and vitreous microRNA (miRNA) signatures of diabetic retinopathy (DR), and then as diagnostic biomarkers for the onset and progression of DR.

公开(公告)号：US20170101642A1

公开(公告)日：2017-04-13

申请号：US15289856

申请日：2016-10-10

申请人： Washington University

发明人： Michael Diamond ,  Rong Zhang

IPC分类号： C12N15/113

CPC分类号： C12N15/1137 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2320/12

摘要： The present invention is directed to compositions targeting the signal peptidase complex and methods of use in treating and preventing flavivirus infection.

公开(公告)号：US09493841B2

公开(公告)日：2016-11-15

申请号：US14658927

申请日：2015-03-16

申请人： Sohail Tavazoie ,  Nils Halberg ,  Kim Png

发明人： Sohail Tavazoie ,  Nils Halberg ,  Kim Png

IPC分类号： C12N15/113 ,  C12Q1/68 ,  C07K16/18 ,  A61K38/17 ,  A61K39/395 ,  A61K39/00

CPC分类号： C12Q1/6886 ,  A61K38/1709 ,  A61K39/3955 ,  A61K45/06 ,  A61K2039/505 ,  A61K2300/00 ,  C07K16/18 ,  C07K2317/73 ,  C07K2317/76 ,  C12N15/113 ,  C12N15/1135 ,  C12N2310/113 ,  C12N2310/141 ,  C12N2320/11 ,  C12N2320/12 ,  C12N2330/31 ,  C12Q2600/106 ,  C12Q2600/118 ,  C12Q2600/136 ,  C12Q2600/158 ,  C12Q2600/16 ,  C12Q2600/178

摘要： This invention concerns pathological angiogenesis and cancer, related treatment methods, and related compositions. Also disclosed are related diagnosis kits and methods.